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WO2001073001A3 - Establishment of cellular manipulations which enhance oligo-mediated gene targeting - Google Patents

Establishment of cellular manipulations which enhance oligo-mediated gene targeting Download PDF

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Publication number
WO2001073001A3
WO2001073001A3 PCT/US2001/009218 US0109218W WO0173001A3 WO 2001073001 A3 WO2001073001 A3 WO 2001073001A3 US 0109218 W US0109218 W US 0109218W WO 0173001 A3 WO0173001 A3 WO 0173001A3
Authority
WO
WIPO (PCT)
Prior art keywords
establishment
relates
mediated gene
gene targeting
oligo
Prior art date
Application number
PCT/US2001/009218
Other languages
French (fr)
Other versions
WO2001073001A2 (en
Inventor
Michael M Seidman
Alokes Majumdar
Original Assignee
Us Health
Michael M Seidman
Alokes Majumdar
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Us Health, Michael M Seidman, Alokes Majumdar filed Critical Us Health
Priority to US10/239,595 priority Critical patent/US7358090B2/en
Priority to AU2001245942A priority patent/AU2001245942A1/en
Publication of WO2001073001A2 publication Critical patent/WO2001073001A2/en
Publication of WO2001073001A3 publication Critical patent/WO2001073001A3/en

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/01—Preparation of mutants without inserting foreign genetic material therein; Screening processes therefor
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y204/00—Glycosyltransferases (2.4)
    • C12Y204/02—Pentosyltransferases (2.4.2)
    • C12Y204/02008—Hypoxanthine phosphoribosyltransferase (2.4.2.8)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3511—Conjugate intercalating or cleaving agent

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  • Genetics & Genomics (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

The invention relates to improved methods for the modification, including recombination, of genes in cells. More specifically, the invention relates to the increased efficiency of modification, including recombination, by introduction of a DNA-modifying molecule into a cell cycle synchronized cell. Additionally, the invention relates to target DNA that has been modified, mutated or marked by the approaches disclosed herein. The invention also relates to cells, tissue, and organisms which have been modified by the invention's methods.
PCT/US2001/009218 2000-03-24 2001-03-22 Establishment of cellular manipulations which enhance oligo-mediated gene targeting WO2001073001A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
US10/239,595 US7358090B2 (en) 2000-03-24 2001-03-22 Establishment of cellular manipulations which enhance oligo-mediated gene targeting
AU2001245942A AU2001245942A1 (en) 2000-03-24 2001-03-22 Establishment of cellular manipulations which enhance oligo-mediated gene targeting

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US19199600P 2000-03-24 2000-03-24
US60/191,996 2000-03-24

Publications (2)

Publication Number Publication Date
WO2001073001A2 WO2001073001A2 (en) 2001-10-04
WO2001073001A3 true WO2001073001A3 (en) 2002-03-21

Family

ID=22707797

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2001/009218 WO2001073001A2 (en) 2000-03-24 2001-03-22 Establishment of cellular manipulations which enhance oligo-mediated gene targeting

Country Status (3)

Country Link
US (1) US7358090B2 (en)
AU (1) AU2001245942A1 (en)
WO (1) WO2001073001A2 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040009602A1 (en) * 2002-05-13 2004-01-15 The Government Of The U.S.A. As Represented By The Secretary Of The Dept. Of Health & Human Services DNA modifying molecules and methods of use thereof
US7947230B2 (en) * 2008-12-16 2011-05-24 Uop Llc Apparatus for regenerating catalyst
CA3014792A1 (en) * 2016-02-16 2017-08-24 Carnegie Mellon University Compositions for enhancing targeted gene editing and methods of use thereof
US20200113821A1 (en) 2017-04-04 2020-04-16 Yale University Compositions and methods for in utero delivery

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1994029461A1 (en) * 1993-06-11 1994-12-22 Commonwealth Scientific And Industrial Research Organisation Method for specific silencing of genes by dna methylation
WO1995001370A1 (en) * 1993-07-02 1995-01-12 Isis Pharmaceuticals, Inc. Higher order structure and binding of peptide nucleic acids
WO1995001364A1 (en) * 1993-06-25 1995-01-12 Yale University Chemically modified oligonucleotide for site-directed mutagenesis
WO1995033493A1 (en) * 1994-06-06 1995-12-14 Research Development Foundation Novel uses of triplex forming oligonucleotides for the treatment of human diseases
WO1996040271A1 (en) * 1995-06-07 1996-12-19 Yale University Treatment of hemoglobinopathies
WO1996040898A1 (en) * 1995-06-07 1996-12-19 Yale University Triple-helix forming oligonucleotides for targeted mutagenesis

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6136601A (en) * 1991-08-21 2000-10-24 Epoch Pharmaceuticals, Inc. Targeted mutagenesis in living cells using modified oligonucleotides
US5604102A (en) * 1992-04-15 1997-02-18 Athena Neurosciences, Inc. Methods of screening for β-amyloid peptide production inhibitors

Patent Citations (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5986053A (en) * 1992-05-22 1999-11-16 Isis Pharmaceuticals, Inc. Peptide nucleic acids complexes of two peptide nucleic acid strands and one nucleic acid strand
WO1994029461A1 (en) * 1993-06-11 1994-12-22 Commonwealth Scientific And Industrial Research Organisation Method for specific silencing of genes by dna methylation
WO1995001364A1 (en) * 1993-06-25 1995-01-12 Yale University Chemically modified oligonucleotide for site-directed mutagenesis
US5962426A (en) * 1993-06-25 1999-10-05 Yale University Triple-helix forming oligonucleotides for targeted mutagenesis
WO1995001370A1 (en) * 1993-07-02 1995-01-12 Isis Pharmaceuticals, Inc. Higher order structure and binding of peptide nucleic acids
WO1995033493A1 (en) * 1994-06-06 1995-12-14 Research Development Foundation Novel uses of triplex forming oligonucleotides for the treatment of human diseases
WO1996040271A1 (en) * 1995-06-07 1996-12-19 Yale University Treatment of hemoglobinopathies
WO1996040898A1 (en) * 1995-06-07 1996-12-19 Yale University Triple-helix forming oligonucleotides for targeted mutagenesis

Also Published As

Publication number Publication date
US7358090B2 (en) 2008-04-15
WO2001073001A2 (en) 2001-10-04
AU2001245942A1 (en) 2001-10-08
US20030211612A1 (en) 2003-11-13

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